後天性視床下部肥満症におけるセメラノチドの試験
2026年7月16日 更新者：Rhythm Pharmaceuticals, Inc.
後天性視床下部肥満患者におけるセトメラノチドの有効性と安全性を評価する第3相、二重盲検、無作為化、プラセボ対照試験
この試験の目的は、セメラノチドが 4 歳以上の後天性視床下部肥満 (HO) 患者の減量、空腹感、および生活の質を改善するためにどのように機能するかを調べることです。
setmelanotide の効果と安全性を判断するために、HO 患者は setmelanotide またはプラセボのいずれかを毎日注射し、最大 60 週間の試験評価を完了します。
調査の概要
研究の種類
介入
入学 (実際)
143
段階
- フェーズ 3
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究場所
-
-
Alabama
-
Birmingham、Alabama、アメリカ、35233
- UAN Pediatric Endocrinology
-
-
California
-
San Diego、California、アメリカ、92123
- Rady Children's Hospital
-
-
Colorado
-
Aurora、Colorado、アメリカ、80045
- Children's Hospital Colorado
-
-
Florida
-
Gainesville、Florida、アメリカ、32610-0296
- University of Florida
-
-
Illinois
-
Chicago、Illinois、アメリカ、60611
- Ann and Robert H. Lurie Children's Hospital
-
-
Iowa
-
Iowa City、Iowa、アメリカ、52242
- University of Iowa Stead Family Department of Pediatrics
-
-
Massachusetts
-
Boston、Massachusetts、アメリカ、02115
- Brigham and Women's Hospital
-
Boston、Massachusetts、アメリカ、02115
- Boston Children's Hospital
-
-
Minnesota
-
Saint Paul、Minnesota、アメリカ、55102
- Children's Minnesota
-
-
New York
-
New York、New York、アメリカ、10032
- Columbia University Irving Medical Center
-
New York、New York、アメリカ、100029
- Icahn School of Medicine at Mount Sinai
-
-
Ohio
-
Columbus、Ohio、アメリカ、43203
- Ohio State Wexner Medical Center
-
-
Oklahoma
-
Oklahoma City、Oklahoma、アメリカ、73122
- Lynn Health Science Institute
-
-
Pennsylvania
-
Philadelphia、Pennsylvania、アメリカ、19104
- Children's Hospital of Philadelphia
-
-
Tennessee
-
Nashville、Tennessee、アメリカ、37232
- Vanderbilt University School of Medicine
-
-
Washington
-
Seattle、Washington、アメリカ、98101
- Seattle Children's Hospital, Research and Foundation - Center for Integrative Brain Research
-
-
-
-
-
Birmingham、イギリス、B46NH
- Birmingham Women and Children's Hospital NHS Trust
-
Hull、イギリス、HU32RW
- Hull University Teaching Hospital
-
London、イギリス、WC1N 1EH
- UCL Great Ormond Street Institute of Child Health
-
-
-
-
-
Utrecht、オランダ、3584CS
- Prinses Maxima Center for Pediatric Oncology
-
-
-
-
Ontario
-
Toronto、Ontario、カナダ、M5G 1X8
- Hospital for Sick Children
-
-
-
-
-
Hamburg、ドイツ、20246
- Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH
-
München、ドイツ、81667
- Medicover Neuroendokrinologie
-
Oldenburg、ドイツ、26133
- University Children's Hospital, Klinikum Oldenburg
-
Ulm、ドイツ、89075
- Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
-
-
-
-
Aichi-ken
-
Nagoya、Aichi-ken、日本、467-8602
- Nagoya City University Hospital
-
-
Nagano
-
Azumino、Nagano、日本、399-8288
- Nagano Children's Hospital
-
-
Tokyo
-
Minato、Tokyo、日本、105-8470
- Toranomon Hospital
-
-
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
4年歳以上 (子、大人、高齢者)
健康ボランティアの受け入れ
いいえ
説明
主な採用基準:
- 後天性視床下部肥満（HO）の文書化された証拠
- 対象年齢 4歳以上
- -視床下部損傷に関連する体重増加と、18歳以上の患者のBMIが30kg / m2以上、または4歳から18歳未満の患者の年齢と性別のBMIが95パーセンタイル以上
- -研究全体および研究後90日間、非常に効果的な避妊方法を使用することに同意する
主な除外基準:
- Prader-Willi症候群（PWS）または低換気、視床下部、自律神経調節不全、神経内分泌腫瘍症候群（ROHHADNET）を伴う急速発症性肥満の診断
- 18歳以上の患者の過去3か月間の体重減少が2%を超えるか、4歳から18歳未満の患者のBMIが2%を超えて減少
- 過去2年以内の肥満手術または処置
- 重度の精神障害の診断;自殺念慮、試み、または行動
- -現在、臨床的に重要な肺、心臓、代謝、または腫瘍性疾患
- -メラノーマまたはプレメラノーマの皮膚病変に関連する重大な皮膚所見（非侵襲的な基底または扁平上皮細胞病変を除く）
- 皮膚がんまたは黒色腫の病歴または近親者の家族歴
- -最初の試験投与前の3か月以内に治験薬/デバイスを使用した臨床試験に参加した
- -以前にセットメラノチドを含む臨床試験に登録したか、セットメラノチドへの以前の暴露
- 1 日 1 回 (QD) の注射レジメンを遵守できない
- 女性、妊娠中および/または授乳中の場合
- -視床下部損傷の前に、他の遺伝的または症候群的状態（例えば、PPL [POMC、PCSK1、LEPR、集合的に]、BBS）に起因する肥満の患者。
- -ホルモン補充療法を受けている場合、投与量はスクリーニング前の少なくとも2か月間安定しています
他のプロトコル定義の包含/除外基準が適用される場合があります。
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：トリプル
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：セテメラノチド
無作為化 2:1 (セメラノチド: プラセボ)
|
毎日の皮下注射のソリューション
他の名前：
|
|
プラセボコンパレーター：プラセボ
無作為化 2:1 (セメラノチド: プラセボ)
|
毎日の皮下注射用に setmelanotide と一致するプラセボ
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
BMI was calculated as weight (kg)/height (m^2).
Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI (≥18 Years of Age) or ≥0.2-point Reduction From Baseline in BMI Z-score (<18 Years of Age)
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
BMI was calculated as weight (kg)/height (m^2).
BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection.
The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package.
A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals).
A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicated an increase in BMI from Baseline.
Baseline was defined as the most recent measurement prior to the first administration of study drug.
Results below represent the percentage of estimated participants with either ≥5% BMI reduction or ≥0.2 point reduction in BMI Z-score by age in each treatment arm as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Percentage of Participants With ≥5% Reduction From Baseline in BMI
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
BMI was calculated as weight (kg)/height (m^2).
Results below represent the percentage of estimated participants with ≥5% BMI reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Change From Baseline in Weekly Average Daily Most Hunger Score in Participants ≥12 Years of Age
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
Change from baseline in hunger scores for participants ≥12 years of age with acquired hypothalamic obesity was evaluated.
Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on an 11-point numeric rating scale.
On Daily Hunger Questionnaire, each of the 2 items (average hunger and most hunger) was scored separately and averaged on weekly basis.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Percentage of Participants (≥12 Years of Age) With a ≥2-point Reduction From Baseline in the Weekly Average Daily Most Hunger Score
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
Hunger score ranged from 0= "not hungry at all" to 10= "hungriest possible" on an 11-point numeric rating scale.
On Daily Hunger Questionnaire, each of the 2 items (average hunger and most hunger) was scored separately and averaged on weekly basis.
Results below represent the percentage of estimated participants ≥12 years of age with ≥2-point reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Change From Baseline in the Weekly Average of the Symptoms of Hyperphagia Composite Score in Participants ≥12 Years of Age
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
Participants ≥12 years of age who were able to self-report were administered the Symptoms of Hyperphagia: Patient (Version 1.0).
The questionnaire consisted of 4 items related to the frequency of a participant's hunger symptoms on a scale (Never, 1 or 2 times, 3 or more times) over the past 24 hours.
The scores on this scale range from 0 to 2 with higher scores indicating more hyperphagia.
Daily composite score was derived as the sum of daily answered questions divided by the number of answered questions per day.
The weekly average of the daily composite scores equals to the sum of daily composite scores divided by the number of days with a composite score within the 7 identified days prior to the visit.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in BMI
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
BMI was calculated as weight (kg)/height (m^2).
Results below represent the percentage of estimated participants with ≥10% BMI reduction in each treatment arm as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Percentage of Participants With a ≥10% Reduction From Baseline in Body Weight
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
Body weight was captured for analysis in kilograms.
Results below represent the percentage of estimated participants with ≥10% reduction in body weight in each treatment arm as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Percent Change From Baseline in Body Weight in Participants ≥18 Years
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
Body weight was captured for analysis in kilograms.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Change From Baseline in BMI Z-Score in Participants <18 Years of Age
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
BMI was calculated as weight (kg)/height (m^2).
BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection.
The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package.
A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals).
A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicates an increase in BMI from Baseline.
Baseline was defined as the most recent measurement prior to the first administration of study drug.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Change From Baseline in Percent of BMI 95th Percentile in Participants <18 Years of Age
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2.
BMI percentile scores are measures of relative weight adjusted for child's age and gender.
The percent of the BMI 95th percentile score expresses the participant's BMI as a percentage of the Centers for Disease Control (CDC) 95th percentile reference population.
Baseline was defined as the most recent measurement prior to the first administration of study drug.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Percentage of Participants <18 Years of Age With ≥0.2-Point Reduction From Baseline in BMI Z-Score
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
BMI was calculated as weight (kg)/height (m^2).
BMI Z-Score calculated for participants <18 years old only is a measure of relative weight adjusted for child's age, sex and height at the time of data collection.
The Z-Scores were calculated using the World Health Organization's WHO 2007 BMI SAS Macro Package.
A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals).
A decrease in BMI Z-score (< 0) indicates a reduction in BMI from Baseline whereas an increase in BMI-Z score (> 0) indicates an increase in BMI from Baseline.
Baseline was defined as the most recent measurement prior to the first administration of study drug.
Results below represent the percentage of estimated participants with ≥0.2 point reduction in BMI Z-score in each treatment arm and the difference between the treatment arms as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Percentage of Participants With BMI <30 kg/m^2 (Aged ≥18 Years) or <95th Percentile (Aged <18 Years) From Baseline
時間枠：After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
BMI was calculated using participant's weight and height assessments, using the following formula: BMI = kg/m^2.
BMI Percentile scores are measures of relative weight adjusted for child's age and gender.
The percent of the BMI 95th percentile score expresses the participant's BMI as a percentage of the CDC 95th percentile reference population.
Baseline was defined as the most recent measurement prior to the first administration of study drug.
Results below represent the percentage of estimated participants with either BMI <30 kg/m^2 (aged ≥18 years) or <95th percentile (aged <18 years) in each treatment arm and the difference between the treatment arms as calculated through the MIANALYZE SAS procedure.
|
After approximately 52 Weeks on a Therapeutic Regimen (baseline up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Change From Baseline in Physical Functioning Score and Total Score on the Impact of Weight on Quality of Life-Lite (IWQOL)
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
The IWQOL-Lite-Clinical Trials (administered to participants ≥18 years of age) is a validated 20-item self-report measure of obesity-specific quality of life questionnaire.
It assessed 2 primary domains of obesity-related health-related quality of life (HRQoL): physical (7 items) and psychosocial (13 items).
Each item was rated on a scale from 0 (worst) to 100 (best), with higher scores indicating better levels of functioning.
It provided composite scores for each domain, as well as a total score, all ranging from 0 (worst) to 100 (best).
Higher scores reflect better levels of functioning and quality of life.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Change From Baseline in Total Score on the Impact of Weight on Quality of Life-Kids (IWQOL-Kids)
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
The IWQOL-Kids (administered to participants between the ages of 11 and <18 years) is a validated 27-item self-report measure of weight-related quality of life for youth.
It provided a total score inclusive of 4 domains: physical comfort, body esteem, social life, and family relations.
Results below represent the total score, which is rated on a scale from 0 (worst) to 100 (best), with higher scores indicating better levels of functioning.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Mean Change From Baseline in Waist Circumference
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
Waist circumference was captured for analysis in centimeters.
LSM and SE were calculated using ANCOVA model.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
|
Pivotal Cohort: Change From Baseline in Systolic and Diastolic Blood Pressure
時間枠：Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
Blood pressure was calculated in millimeters of mercury.
|
Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)
|
協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
捜査官
- スタディチェア：David Meeker, MD、Rhythm Pharmaceuticals, Inc.
出版物と役立つリンク
研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。
一般刊行物
- Miller JL, van Santen HM, Phillips SA, Hamilton J, Aberle J, Sathyapalan T, Mohamed Z, McCormack SE, Shoemaker AH, Kelsey MM, Ghalib L, Stalla G, Thaker VV, Habiby R, Ode KL, Wabitsch M, Dattani M, Abuzzahab MJ, Abdullatif H, Morgan R, Stefater-Richards M, Aroda VR, Friedrich C, Han JC, Ono H, Nagasaki K, Tanaka T, Isojima T, Arima H, Scimia C, Yuan G, Muller HL, Roth CL; TRANSCEND Trial Group. Setmelanotide for the Treatment of Acquired Hypothalamic Obesity. N Engl J Med. 2026 Jul 9;395(2):138-150. doi: 10.1056/NEJMoa2512275.
- Roth CL, Phillips SA, McCormack SE, Ode KL, Kelsey MM, Liu J, Ervin C, Mallya UG, Norcross L, Miller JL. Quality of life improvements with setmelanotide treatment in acquired hypothalamic obesity: TRANSCEND trial interview results from US participants. Front Behav Neurosci. 2026 Jul 15;20:1830070. doi: 10.3389/fnbeh.2026.1830070. eCollection 2026.
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (実際)
2023年4月26日
一次修了 (実際)
2025年3月18日
研究の完了 (推定)
2027年4月16日
試験登録日
最初に提出
2023年3月1日
QC基準を満たした最初の提出物
2023年3月16日
最初の投稿 (実際)
2023年3月20日
学習記録の更新
投稿された最後の更新 (実際)
2026年8月10日
QC基準を満たした最後の更新が送信されました
2026年7月16日
最終確認日
2026年7月1日
詳しくは
本研究に関する用語
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- RM-493-040
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
いいえ
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
はい
米国FDA規制機器製品の研究
いいえ
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。